acth treatment of apol1- associated nephropathy

Conditions: Kidney Disease

Summary: the purpose of this research study is to determine if the study drug h.c. acthar gel slows the progression of your kidney disease. this drug is a steroid-based medicine with fewer side effects than other steroids used for treatment of kidney diseases similar to apol1 nephropathy.


Eligibility criteria:
inclusion criteria: * non-diabetic african-american with two apol1-risk genotypes * age ≥21 years * bmi \< 40 kg/m2 * hemoglobin a1c \<6.5% * egfr ≥30 ml/min/1.73m2 * historical urine protein: creatinine ratio ≥ 1.0 g/g * strong clinical suspicion of apol1-associated nephropathy or history of biopsy proven focal segmental glomerulosclerosis (fsgs) or focal global glomerulosclerosis (fggs) * women of childbearing potential: negative serum pregnancy test at screening and agreement to follow a medically acceptable form of contraception for the duration of acthar administration and 4 weeks thereafter exclusion criteria: * diagnosis of diabetes mellitus and/or on pharmacologic treatment for diabetes * medical condition that could cause secondary fsgs * history of sensitivity to steroids (psychosis, steroid-induced diabetes) * chronic systemic corticosteroid use (prednisone or equivalent systemic steroid taken for more than 4 consecutive weeks within 6 months prior to screening). intra-articular, inhaled, and topical steroids are not exclusion criteria. * contraindication to acthar per package insert: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery (within previous 6 months), history of or the presence of peptic ulcer (within 6 months prior to screening), adrenal insufficiency or hyperfunction. * acute glaucoma diagnosed ≤3 months prior to screening * biopsy proven glomerular disease other than fsgs or fggs * live or live attenuated vaccine received within 1 month prior to screening, or planned administration once enrolled in the study * uncontrolled hypertension (htn) (≥ 180/110 mmhg) and frequent admissions (≥1 admission per 6 months interval) for hypertensive urgency or hypertensive emergency * unstable cardiovascular disease: history of congestive heart failure (nyha functional class iii-iv); history of dilated cardiomyopathy with ejection fraction \< 40%; any of the following events within 3 months of screening: unstable angina, myocardial infarction, coronary artery bypass graft or percutaneous transluminal coronary angioplasty, transient ischemic attack or cerebrovascular accident, unstable arrhythmia * uncontrolled volume overload: history of moderate or severe peripheral edema; on loop diuretics ≥ 120 mg daily of furosemide or ≥ 3.0 mg daily of bumetanide or ≥ 150 mg daily of ethacrynic acid or ≥ 60 mg daily of torsemide; * history of secondary hypertension (i.e., renal artery stenosis, primary aldosteronism or pheochromocytoma) * significant comorbidities (e.g., advanced malignancy, advanced liver disease) with a life expectancy of less than 1 year * subject is expected to initiate dialysis within 6 months * previous treatment on a drug being investigated for the treatment of fsgs * known diagnosis of human immunodeficiency virus, hepatitis b, or hepatitis c * known history of a primary immunodeficiency or an underlying condition such as splenectomy that predisposes the subject to infections * systemic hematologic disease (e.g., hematologic malignancy, sickle cell anemia, myelodysplastic syndrome) * current malignancy or history of malignancy within 5 years of screening, with the exception of non-melanoma skin cancers and cervical intraepithelial neoplasia * treatment for any malignancy (e.g., radiation, chemotherapy, hormone therapy, or biologics) within 5 years of screening, with the exception of locally excised non-melanoma skin cancer or cervical intraepithelial neoplasia * pregnant or breast feeding, or might become pregnant during the study or within 4 weeks after the end of treatment * female of reproductive potential not willing to use highly effective methods of birth control during treatment and for 4 weeks after the end of treatment * currently receiving systemic antibiotics for treatment of an active infection; or history of frequent infections (more than one event per 6 months) * history of any organ transplant * bipolar disorder, or major depressive disorder characterized by severe depression requiring hospitalization, or history of suicidal ideation/attempts * currently enrolled in another interventional study, or less than 4 weeks since ending another interventional study(s) or receiving investigational agents(s) * subject has a disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or comply with all required study procedures.

Keywords: africanamericans hypertension proteinuria apol1 gene